Active Studies

Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)

·         Study Status: Open to enrollment

·         Patient Population: DM1

·         Participation Duration: 24 months

·         Active Sites:

o   Ludwig Maximilian University of Munich

o   NEuroMuscular Omnicentre

o   Radboud University

o   Stanford University

o   Virginia Commonwealth University

o   University College London

o   The University of Auckland

o   University of California, Los Angeles

o   University of Colorado

o   University of Florida Health

o   University of Iowa

o   University of Kansas Medical Center

o   University of Rochester Medical Center

o   St. George’s, University of London

o   Université de Sherbrooke

o   University of California, San Diego

Upcoming Sites:

o   Osaka University

o   UT Health San Antonio

o   University of Sheffield

·         Study Summary:

This is a non-interventional study observing the natural progression of DM1 in adult patients over the course of 24 months. The study’s primary goal is to identify standardized clinical outcome assessments, in the form of functional measures, to hasten therapeutic development.

Assessing Pediatric Endpoints in DM1 (ASPIRE-DM1)

·         Study Status: Open to enrollment

·         Patient Population:

o   CDM

o   ChDM

·         Participant Duration: 18 months

·         Active Sites:

o   Virginia Commonwealth University

o   NEuroMuscular Omnicentre

o   University of California, Los Angeles

o   University of Kansas Medical Center

o   University of Rochester Medical Center

·         Study Summary:

This is a non-interventional study observing the natural progression of CDM and ChDM in patients over the course of 18 months. The study’s primary goal is to evaluate appropriate measures of physical function, cognitive function, and quality of life in children with CDM and ChDM.